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Oral complement factor D inhibitor danicopan for paroxysmal nocturnal hemoglobinuria. 口服补体因子 D 抑制剂达尼可潘治疗阵发性夜间血红蛋白尿。
IF 4.4 3区 医学 Q2 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-11 DOI: 10.1080/17512433.2024.2403638
Bo Xu,Jiecan Zhou
INTRODUCTIONParoxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder characterized by episodic hemolysis, with additional clinical manifestations including thrombosis and bone marrow failure. The US FDA approved a complement factor D inhibitor, danicopan (Voydeya™), previously known as ACH-4471, for the treatment of extravascular hemolysis in adults with PNH on March 29, 2024. The primary purpose of this review is to examine the clinical efficacy and safety of danicopan.AREAS COVEREDWe systematically searched for articles on PubMed, Web of Science, and three publishers Springer, Elsevier, Wiley up to May 6, 2024.EXPERT OPINIONDanicopan acts on the alternative pathway of the complement cascade, preferentially controlling C3 fragment-mediated extravascular hemolysis. Recommended dosage is 150 mg orally three times a day, which can be increased to 200 mg three times a day when necessary. Vaccination is required before administration to prevent infections by encapsulated bacteria. In a pivotal phase 3 trial ALPHA, danicopan significantly increased hemoglobin levels compared to placebo (P<0.0001), 60% of patients experienced an increase in hemoglobin levels of at least 2 g/dL, compared to none in the placebo group (adjusted difference of 47%; P<0.0001). Common adverse events during danicopan treatment include headache and upper respiratory tract infection.
引言 阵发性夜间血红蛋白尿症(PNH)是一种罕见的血液病,以偶发性溶血为特征,并伴有血栓形成和骨髓衰竭等其他临床表现。美国 FDA 于 2024 年 3 月 29 日批准了一种补体因子 D 抑制剂 Danicopan (Voydeya™)(以前称为 ACH-4471),用于治疗成人 PNH 患者的血管外溶血。本综述的主要目的是研究达尼可潘的临床疗效和安全性。涵盖领域我们系统地检索了PubMed、Web of Science以及Springer、Elsevier、Wiley三家出版社截至2024年5月6日的文章。专家观点达尼可潘作用于补体级联的替代途径,优先控制C3片段介导的血管外溶血。建议剂量为口服 150 毫克,每天三次,必要时可增至 200 毫克,每天三次。用药前需接种疫苗,以防止被包裹的细菌感染。在一项关键性的 3 期试验 ALPHA 中,与安慰剂相比,达尼考潘可显著提高血红蛋白水平(P<0.0001),60% 的患者血红蛋白水平至少提高了 2 g/dL,而安慰剂组患者的血红蛋白水平没有提高(调整后差异为 47%;P<0.0001)。达尼可潘治疗期间常见的不良反应包括头痛和上呼吸道感染。
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引用次数: 0
Association between sodium-glucose cotransporter 2 inhibitors and eye disorders: a systematic review and meta-analysis. 钠-葡萄糖共转运体 2 抑制剂与眼疾之间的关系:系统回顾和荟萃分析。
IF 3.6 3区 医学 Q2 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-11 DOI: 10.1080/17512433.2024.2399073
Bo Xu, Bo Kang, Fan Tang, Jiecan Zhou, Zunbo He

Introduction: Lowering blood glucose is important to prevent long-term microvascular complications in adults with type 2 diabetes mellitus (T2DM). Various evidence suggests that sodium-glucose cotransporter 2 (SGLT2) inhibitors are beneficial for microvascular diseases. This study was designed to investigate whether SGLT2 inhibitors have an effect on eye disorders.

Methods: We searched PubMed, Web of Science, and ClinicalTrials.gov for randomized, double-blind, placebo-controlled trials with at least 24 weeks of follow-up up to 20 December 2023. Mantel-Haenszel statistical method, risk ratios (RRs) and 95% confidence intervals (CIs) were used to analyze the binary variables.

Results: We included a total of 40 studies covering 104,586 participants. T2DM was present in 84.5% of the subjects. SGLT2 inhibitors had no significant effect on overall eye events compared to placebo (RR 0.99; 95%CI 0.86-1.15; p = 0.91), nor did subgroup analysis. We did not observe significant heterogeneity (I2 = 0; p = 0.99). Analysis of all secondary outcomes showed that SGLT2 inhibitors did not cause a significantly different effect from placebo. Meta-analysis in the entire T2DM population showed results consistent with those in the overall population.

Conclusion: SGLT2 inhibitors did not have a significant effect on eye disorders during treatment, regardless of baseline conditions and duration of treatment.

简介降低血糖对于预防成人 2 型糖尿病(T2DM)患者的长期微血管并发症非常重要。各种证据表明,钠-葡萄糖共转运体 2(SGLT2)抑制剂对微血管疾病有益。本研究旨在探讨 SGLT2 抑制剂是否对眼部疾病有影响:我们在PubMed、Web of Science和ClinicalTrials.gov上检索了截至2023年12月20日至少随访24周的随机、双盲、安慰剂对照试验。采用曼特尔-海恩泽尔统计方法、风险比(RR)和95%置信区间(CI)分析二元变量:我们共纳入了 40 项研究,覆盖 104 586 名参与者。84.5%的受试者患有 T2DM。与安慰剂相比,SGLT2 抑制剂对总体眼部事件没有显著影响(RR 0.99;95%CI 0.86-1.15;P = 0.91),亚组分析也是如此。我们没有观察到明显的异质性(I2 = 0;P = 0.99)。对所有次要结果的分析表明,SGLT2 抑制剂与安慰剂的效果没有明显差异。对所有 T2DM 患者进行的 Meta 分析表明,结果与整个人群的结果一致:结论:无论基线条件和疗程长短如何,SGLT2 抑制剂在治疗期间都不会对眼部疾病产生显著影响。
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引用次数: 0
Correction. 更正。
IF 3.6 3区 医学 Q2 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-08 DOI: 10.1080/17512433.2024.2402165
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引用次数: 0
Drug related adverse event assessment in neonates in clinical trials and clinical care. 在临床试验和临床护理中对新生儿进行药物相关不良事件评估。
IF 3.6 3区 医学 Q2 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-01 Epub Date: 2024-08-13 DOI: 10.1080/17512433.2024.2390927
Nadir Yalcin, John van den Anker, Samira Samiee-Zafarghandy, Karel Allegaert

Introduction: Assessment of drug-related adverse events is essential to fully understand the benefit-risk balance of any drug exposure, weighing efficacy versus safety. This is needed for both drug labeling and clinical decision-making. Assessment is based on seriousness, severity and causality, be it more difficult to apply in neonates. Adverse event detection or prevention in the neonatal clinical setting is also more complicated because of polypharmacy, and off-label or unlicensed pharmacotherapy.

Areas covered: Tools became available to assess severity and causality of adverse events in neonates recruited in clinical trials. The first version of the Neonatal Adverse Event severity score (NAESS) reduced the inter-observer variability. Causality tools like the Naranjo score were also tailored to neonates. These tools are also instrumental to support proactive pharmacovigilance in clinical care, while multidisciplinary care teams and computerized pharmacovigilance using advanced data analysis, like machine learning are emerging approaches to develop effective decision strategies.

Expert opinion: All stakeholders involved in development of medicines or its clinical use should be aware of the limitations of the currently available assessment tools. Extension and optimization of these tools, advanced data analysis approaches, and capturing the variability in time-dependent physiology are warranted to improve pharmacovigilance in neonates.

导言:评估与药物相关的不良事件对于充分了解任何药物暴露的效益与风险平衡、权衡疗效与安全性至关重要。这对于药物标签和临床决策都是必需的。评估以严重性、严重程度和因果关系为基础,但在新生儿中较难应用。新生儿临床环境中的不良事件检测或预防工作也更加复杂,因为新生儿使用多种药物、标签外或无证药物治疗:目前已有工具可用于评估临床试验中新生儿不良事件的严重性和因果关系。第一版新生儿不良事件严重程度评分(NAESS)减少了观察者之间的差异。纳兰霍评分等因果关系工具也是为新生儿量身定制的。这些工具还有助于支持临床护理中的前瞻性药物警戒,而多学科护理团队和使用先进数据分析(如机器学习)的计算机化药物警戒则是制定有效决策策略的新兴方法:专家意见:所有参与药品研发或临床使用的相关人员都应认识到现有评估工具的局限性。为了改善新生儿的药物警戒,有必要对这些工具进行扩展和优化,采用先进的数据分析方法,并捕捉随时间变化的生理变化。
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引用次数: 0
The evolution of clinical trials for pediatric pulmonary hypertension: are the needs of patients and their caregivers being met? 小儿肺动脉高压临床试验的演变:是否满足了患者及其护理人员的需求?
IF 3.6 3区 医学 Q2 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-01 DOI: 10.1080/17512433.2024.2396119
Julie Wacker, Raphael Joye, Leon Genecand, Frederic Lador, Maurice Beghetti

Introduction: Pediatric pulmonary hypertension is a rare condition. Survival remains poor in the current management era. There is a lack of data regarding the medical management of pediatric pulmonary hypertension and most pulmonary vasodilators are used off-label in children.

Areas covered: Pediatric pulmonary hypertension clinical trials' design and realization face many hurdles, including poor recruitment, limited available pharmacologic and physiologic data in children of various ages, ethical issues, and the lack of validated trial endpoint. Innovative clinical trial designs have emerged and may allow us to overcome some of these issues. Extrapolation of adult data to children, with additional pharmacokinetic and safety data, remains extremely important and valid in etiologies where the pediatric and the adult pathophysiologies are believed to be similar.

Expert opinion: Close collaboration between sponsors, regulators, patients, caregivers, physicians and researchers is necessary to develop efficacious and safe drugs for pediatric pulmonary hypertension. The increasing involvement of patients' and caregivers' participation in the development of clinical trials should help shape future research that is feasible and meaningful to the patients.

简介小儿肺动脉高压是一种罕见病。在目前的治疗时代,存活率仍然很低。有关小儿肺动脉高压医疗管理的数据匮乏,大多数肺血管扩张剂在儿童中的使用都是非标的:小儿肺动脉高压临床试验的设计和实现面临许多障碍,包括招募不足、不同年龄儿童的药物和生理数据有限、伦理问题以及缺乏有效的试验终点。创新的临床试验设计已经出现,或许能让我们克服其中的一些问题。将成人数据外推至儿童,并提供更多的药代动力学和安全性数据,对于认为儿童和成人病理生理学相似的病因仍极为重要和有效:赞助商、监管机构、患者、护理人员、医生和研究人员之间的紧密合作对于开发有效、安全的小儿肺动脉高压药物十分必要。患者和护理人员越来越多地参与到临床试验的开发过程中,这应有助于形成对患者可行且有意义的未来研究。
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引用次数: 0
Sodium-glucose co-transporter 2 inhibition and acute myocardial infarction: the DAPA-MI and EMPACT-MI trials. 钠-葡萄糖共转运体 2 抑制与急性心肌梗死:DAPA-MI 和 EMPACT-MI 试验。
IF 3.6 3区 医学 Q2 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-01 Epub Date: 2024-08-12 DOI: 10.1080/17512433.2024.2390921
Muhammad Adnan Zaman, Sidra Kalsoom
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引用次数: 0
The outcomes of goal-oriented medication reviews for the elderly: current research and future directions. 以目标为导向的老年人用药审查结果:当前研究与未来方向。
IF 3.6 3区 医学 Q2 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-01 Epub Date: 2024-09-03 DOI: 10.1080/17512433.2024.2400248
Janet Sultana, Graziella Aquilina, Malak Bashir Lagaa, Nicole Micallef, Janet Mifsud
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引用次数: 0
Hydroxyurea in the sickle cell disease modern era. 镰状细胞病时代的羟基脲。
IF 3.6 3区 医学 Q2 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-01 Epub Date: 2024-08-20 DOI: 10.1080/17512433.2024.2390915
Chazmyn Riley, Walter K Kraft, Robin Miller

Introduction: Sickle cell disease is an inherited disorder characterized by hemoglobin S polymerization leading to vaso-occlusion and hemolytic anemia. These result in a variety of pathological events, causing both acute and chronic complications. Millions around the world are affected by sickle cell disease with predominance in sub-Saharan Africa. Hydroxyurea was the first drug approved for use in sickle cell disease to reduce the occurrence of painful crises and blood transfusions in patients with frequent, moderate to severe painful crises.

Areas covered: With the development of new therapeutics, the role of hydroxyurea is evolving. This narrative review aims to provide clinical data, safety information, and supplementary evidence for the role of hydroxyurea in the current era of sickle cell disease. A comprehensive literature search of databases, including PubMed and Cochrane Library, was conducted from 1963 to 2024.

Expert opinion: Even though new medications have been approved for sickle cell disease, hydroxyurea remains the gold standard. Hydroxyurea is not only a disease modifier but it has additional clinical benefits, it is affordable, and its longevity has prompted expanded research in areas such as underutilization and pharmacogenomics. As the treatment landscape evolves, hydroxyurea's long-standing record of efficacy and safety continues to support its role as a key agent in disease management.

简介镰状细胞病是一种遗传性疾病,其特点是血红蛋白 S 聚合导致血管闭塞和溶血性贫血。镰状细胞病会导致各种病理现象,引起急性和慢性并发症。全世界有数百万人受到镰状细胞病的影响,其中以撒哈拉以南非洲地区居多。羟基脲是第一种获准用于镰状细胞病的药物,可减少疼痛危象的发生,并减少经常出现中度至重度疼痛危象的患者的输血:随着新疗法的开发,羟基脲的作用也在不断变化。这篇叙述性综述旨在为羟基脲在当前镰状细胞病时代的作用提供临床数据、安全性信息和补充证据。我们对包括 PubMed 和 Cochrane 图书馆在内的数据库进行了全面的文献检索,时间跨度为 1963 年至 2024 年:专家意见:尽管镰状细胞病的新药已获批准,但羟基脲仍是金标准。羟基脲不仅是一种疾病调节剂,还具有额外的临床益处,而且价格低廉,其长效性促使人们在未充分利用和药物基因组学等领域扩大研究。随着治疗领域的不断发展,羟基脲长期以来的有效性和安全性记录将继续支持其作为疾病管理的关键药物。
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引用次数: 0
Emerging pharmacological options in the treatment of idiopathic pulmonary fibrosis (IPF). 治疗特发性肺纤维化(IPF)的新药物选择。
IF 3.6 3区 医学 Q2 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-01 Epub Date: 2024-08-27 DOI: 10.1080/17512433.2024.2396121
Katyayini Aribindi, Gabrielle Y Liu, Timothy E Albertson

Introduction: Idiopathic pulmonary fibrosis (IPF) is a progressive-fibrosing lung disease with a median survival of less than 5 years. Currently, two agents, pirfenidone and nintedanib are approved for this disease, and both have been shown to reduce the rate of decline in lung function in patients with IPF. However, both have significant adverse effects and neither completely arrest the decline in lung function.

Areas covered: Thirty experimental agents with unique mechanisms of action that are being evaluated for the treatment of IPF are discussed. These agents work through various mechanisms of action, these include inhibition of transcription nuclear factor k-B on fibroblasts, reduced expression of metalloproteinase 7, the generation of more lysophosphatidic acids, blocking the effects of transforming growth factor ß, and reducing reactive oxygen species as examples of some unique mechanisms of action of these agents.

Expert opinion: New drug development has the potential to expand the treatment options available in the treatment of IPF patients. It is expected that the adverse drug effect profiles will be more favorable than current agents. It is further anticipated that these new agents or combinations of agents will arrest the fibrosis, not just slow the fibrotic process.

简介特发性肺纤维化(IPF)是一种进行性纤维化肺病,中位生存期不到5年。目前,有两种药物(吡非尼酮和宁替达尼)被批准用于治疗这种疾病,这两种药物都被证明可以降低IPF患者肺功能的下降速度。然而,这两种药物都有明显的不良反应,而且都不能完全阻止肺功能的下降:讨论了三十种具有独特作用机制的实验性药物,这些药物正在被评估用于治疗 IPF。这些药物通过不同的作用机制发挥作用,其中包括抑制成纤维细胞上的转录核因子k-B、减少金属蛋白酶7的表达、生成更多溶血磷脂酸、阻断转化生长因子ß的作用以及减少活性氧,这些都是这些药物一些独特作用机制的例子:新药开发有可能扩大治疗 IPF 患者的选择范围。预计新药的不良反应将比现有药物更为有利。此外,预计这些新药或药物组合将阻止纤维化,而不仅仅是减缓纤维化进程。
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引用次数: 0
OPTIMA-ID: development and validation of a medicine optimization tool for older adults with intellectual disability. OPTIMA-ID:开发和验证针对智障老年人的药物优化工具。
IF 3.6 3区 医学 Q2 PHARMACOLOGY & PHARMACY Pub Date : 2024-09-01 Epub Date: 2024-08-13 DOI: 10.1080/17512433.2024.2390913
Juliette O'Connell, Ashleigh Gorman, Éilish Burke, Shoumitro Deb, Martin Charles Henman, Philip McCallion, Mary McCarron, Tara Mullally, Niamh Mulryan, Máire O'Dwyer, Cristín Ryan

Background: Older people (i.e. ≥40 years) with intellectual disability have unique medication needs and may experience high levels of potentially inappropriate prescribing. Despite the availability of tools to optimize older adults' prescriptions, there is no comprehensive tool specifically for use in older adults with intellectual disability. We aimed to develop a tool for this purpose: Optimizing Pharmaco-Therapy and Improving Medication for Ageing with Intellectual Disability (OPTIMA-ID).

Research design and methods: A draft tool was developed based on literature review and clinical expertise. Focus groups with healthcare professionals and people with intellectual disability were conducted to refine the tool. The tool was presented electronically to an expert panel for Delphi validation. Median level of agreement and 75th percentile values were used to establish if consensus was reached. Criteria were accepted, rejected, revised or removed to develop the final tool.

Results: Following two Delphi rounds, consensus on the content of OPTIMA-ID was reached for 67 prescribing criteria, 63 of which were agreed upon after Round 1 and a further 4 criteria accepted after Round 2.

Conclusions: OPTIMA-ID contains 67 criteria that can optimize medications for older people with intellectual disability. Its effectiveness, feasibility and impact on patient outcomes need to be established.

背景:智障老年人(即年龄≥40 岁)有独特的用药需求,可能会出现大量潜在的不当处方。尽管目前已有一些优化老年人处方的工具,但还没有专门用于智障老年人的综合工具。我们旨在为此开发一种工具:智障老年人优化药物治疗和改善用药(OPTIMA-ID):研究设计与方法:根据文献综述和临床专业知识开发了工具草案。为完善该工具,与医护人员和智障人士开展了焦点小组讨论。该工具以电子形式提交给专家小组进行德尔菲验证。采用协议中值和第 75 百分位值来确定是否达成共识。对标准进行接受、拒绝、修改或删除,以制定最终工具:经过两轮德尔菲验证,就 67 项处方标准达成了共识,其中 63 项标准在第一轮验证后达成一致,另有 4 项标准在第二轮验证后被接受:结论:OPTIMA-ID 包含 67 项标准,可优化智障老年人的用药。结论:OPTIMA-ID 包含 67 项可优化智障老年人用药的标准,其有效性、可行性和对患者预后的影响有待确定。
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引用次数: 0
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Expert Review of Clinical Pharmacology
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